Gene-editing therapy aimed at two targets—HIV-1, the virus that causes AIDS, and CCR5, the co-receptor that helps the virus get into cells—can effectively eliminate HIV infection, new research from the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center shows. The study, published online in the journal, is the first to combine a dual gene-editing strategy with antiretroviral drugs to cure animals of HIV-1.
"We are true partners, and what we achieved here is really spectacular," Dr. Gendelman said."Dr. Khalili's team generated the essential gene-editing constructs, and we then applied those constructs in our LASER-ART mouse model at Nebraska, figuring out when to administer gene-editing therapy and carrying out analyses to maximize HIV-1 excision, CCR5 inactivation, and suppression of viral growth.
To prevent rebound infection, Dr. Khalili and colleagues began work on next-generation CRISPR technology for HIV excision, developing a new, dual system aimed at permanently eliminating HIV from the animal model."From success stories of human HIV patients who have undergone bone marrow transplantation for leukemia and been cured of HIV, our hypothesis was that the loss of the virus's receptor, CCR5, is important to permanently eliminating HIV infection," he explained.